U.S FDA Approves AQVESME™ (mitapivat) for Adults with Thalassemia
On December 23, 2025, the U.S. Food and Drug Administration (FDA) approved AQVESME™ (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia. This approval marks a significant milestone for the thalassemia community, as AQVESME is now the only FDA-approved medicine indicated for anemia in both non-transfusion-dependent and transfusion-dependent thalassemia.
AQVESME is an oral pyruvate kinase (PK) activator developed by Agios Pharmaceuticals. With this approval, AQVESME will be marketed in the U.S. specifically for the thalassemia indication, while mitapivat will continue to be marketed as PYRUKYND® for pyruvate kinase deficiency.
“This approval represents an important step forward for individuals with thalassemia,” said Hanny Al-Samkari, M.D., an investigator in the Phase 3 clinical program. Results from the ENERGIZE and ENERGIZE-T trials showed that AQVESME helped address anemia, fatigue, and transfusion burden—some of the most significant challenges faced by people living with the disease.
The FDA approval is based on data from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials, which enrolled 452 adults representative of the real-world thalassemia population. Both studies met all primary and key secondary endpoints, demonstrating significant improvements in hemoglobin levels, fatigue, quality of life measures, and reductions in transfusion burden compared to placebo.
Ralph Colasanti, National President of Cooley’s Anemia Foundation, called the approval “an important new treatment option for the thousands of adults living with thalassemia in the U.S., including those with non-transfusion-dependent thalassemia who previously had no approved alternatives.”
In clinical trials, a small number of patients experienced adverse reactions suggestive of hepatocellular injury. As a result, AQVESME will be available only through the AQVESME REMS (Risk Evaluation and Mitigation Strategy) program, which includes required liver monitoring and education for patients and healthcare providers.
Agios expects AQVESME to be available in the U.S. in late January 2026, following implementation of the REMS program.